Treatment Options

Our team of dermatologists specializes in caring for children with epidermolysis bullosa (EB) and are on the very cutting edge of treatments. We are the first in the world to develop a gene therapy skin graft to help children with EB grow new, healthy skin. As a respected national referral center for EB and other rare genetic skin disorders, we give children hope to live with less pain than ever before.

In our dedicated epidermolysis bullosa clinic, we meet families from many different states across the United States, as well as other countries, for expert opinions, and we provide lifelong care for children with the most challenging and complex EB.

Gene therapy skin grafts (pz-cel therapy)

Our doctors are also researchers, and for decades they have been studying how to improve and/or cure severe dystrophic epidermolysis bullosa (a specific type of EB) with gene therapy, so that one day no child will have to suffer without hope. We helped develop a first-of-its-kind therapeutic advancement.

  • Via clinical trials and FDA approval, we can now use your child’s own skin cells to grow genetically engineered sheets of skin.
  • We obtain skin cells from your child by performing a small skin biopsy, and then insert normal collagen VII genes. Once the skin sheets grow, we surgically graft them onto your child’s large, nonhealing wounds in the operating room.
  • Your child will need to stay in the hospital for approximately five to 10 days after the procedure to ensure proper recovery.
  • As their new skin grows, the wound heals. Our studies on pz-cel therapy achieved at least 50% wound healing in more than 80% of the wounds treated.
  • Because the skin grafts are created from your child’s own cells, their immune system does not reject the grafts.
  • Up to 12 pieces of engineered grafts can be applied each time to treat many areas of the body.
  • We were the first in the world to develop this type of gene therapy for dystrophic EB, and we are the only hospital west of the Rocky Mountains to offer pz-cel treatment to children with dystrophic EB, and one of the three worldwide, all located in the U.S.
  • Our skin graft treatment (pz-cel) has reduced children’s pain and lowered their risk for developing skin cancer. It also enables them to sleep more comfortably, attend school, and function in daily activities with more ease.

Gene therapy topical gel

Our clinician scientists have also played a pivotal role in developing another topical gene therapy for children with epidermolysis bullosa. The stable FDA-approved gel is applied topically during regular bandage changes each week to treat smaller wounds.

  • The gel delivers missing normal collagen VII genes, which help make the skin stronger and less prone to breaking. As wounds and blisters heal, the treatment helps relieve your child’s pain.
  • The genetically modified gel can be used on infants above 6 months of age with dystrophic EB.
  • The gel can be administered in clinic or at home by a certified care provider.

Whole-child care

If your child has severe EB, they need care from many specialists. We support your child, and your family, by integrating nutritionists, physical and occupational therapists, and social workers, as well as many other subspecialities, including ear, nose, and throat, hematology, gastroenterology, orthopedic surgery, and others, into our care to give your child the best chance for a good quality of life.

Our team of providers also supports parents in managing daily care, including learning how to wrap dressings, prevent infections, and alleviate pain and itching.